ASGCT Congressional Briefing: Gene Therapy for Ultrarare and N-of-1 Diseases

ASGCT hosted a Congressional briefing on August 3, 2026 focused on cell and gene therapy (CGT) for ultrarare and N-of-1 genetic diseases. The event brought together academic researchers, industry leaders, and patient advocates to explore how CGT and platform-based development are creating new possibilities for patients who currently have no available treatment options. Learn more about ASGCT's policy and advocacy work at asgct.org/advocacy. For patient education resources, visit patienteducation.asgct.org.